About 15,000 Americans are afflicted with Duchenne Muscular Dystrophy, the most common type of muscular dystrophy.¹ DMD is caused by the absence of the dystrophin protein that is essential to keeping muscle cells intact, resulting in the gradual loss of the ability to perform activities independently.¹ It generally affects boys, about one in every 3600 male infants, and is often seen in people without a family history of the affliction.² On February 9th, the Food and Drug Administration approved the drug Emflaza (deflazacort) to treat DMD. According to the FDA, the approval marks the first corticosteroid, a synthetically produced hormone analog that reduces inflammation, available in the United States to treat the disease.

The drug’s market price in the United States is $89,000 per year.³ The Chief Financial Officer of Marathon Pharmaceuticals, the company that produces Emflaza, said in an interview that after rebates and discounts, the net price would come down to $54,000. ² However, the drug has been widely available in other countries for decades. According to CNN, the drug costs $1,200 abroad annually.³ The company sought the “orphan drug” approvals process which is a special pathway that seeks to encourage the development of drugs for very rare diseases.¹ With this label, the company is granted exclusive rights to sell the drug for seven years.² The generic version of Emflaza has long been available outside of the United States. Many physicians argue that gaining federal approval for the “orphan drug” designation has important advantages, one of which being insurance coverage.¹

Parent Project Muscular Dystrophy, a prominent patient advocacy group, said in a statement that the organization was excited about the FDA approval and hopes that it will result in easier access for more families. The group added, “There are many questions still to be answered, including pricing, and we look forward to talking to Marathon with the community to begin to answer those questions.”² In response to backlash over the drug’s price, Marathon announced a halt on the commercial launch of Emflaza. During this time, families can still purchase the drug from foreign pharmaceutical companies. However, the FDA has indicated that once the drug hits the market in the United States, the foreign option will no longer be available.³ The drug’s price tag has also prompted bipartisan Congressional disapproval. Republican Senator Chuck Grassley of the Senate Judiciary Committee announced the opening of an inquiry into potential conflicts with the Orphan Drug Act. Senator Bernie Sanders (I-VT) and Elijah Cummings (D-MD) sent a letter to Marathon calling the price “unconscionable.”⁴

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